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Foriel 2015 Abstract MiP2015
Additional label MiP2015  +
Enzyme Complex I  +
Event A3  + , Poster  + , P-flash  +
Has abstract Among the wide range of mitochondrial diso
Among the wide range of mitochondrial disorders, defects in the oxidative phosphorylation (OxPhos) are the most prevalent. OxPhos deficiencies often lead to early death and are associated with severe and highly variable clinical symptoms. Despite intense efforts in the comprehension of the mechanisms underlying mitochondrial disorders, patients are still without effective treatment. The need of predictive ''in vivo'' models of the pathology is an important issue in the development of new therapeutics in order to study their therapeutic potential, toxicity and pharmacokinetics. Due to the extreme genetic and phenotypic heterogeneity of OxPhos disorders one cannot rely on a single in ''vivo model''. Here we present the method and strategy we use to create, characterize and validate a set of ''Drosophila melanogaster'' models of nuclear DNA-encoded OxPhos subunits and preliminary results of systematic evaluation of Khondrion´s lead compound. We primarily focus on complex I by knocking down the core and accessory subunits the most prone to mutation in patients and selecting phenotypes-readouts suitable for drug screening (death at critical stages of development, survival curves, ROS level). These models will represent a valuable tool with predictive power to evaluate new potential therapeutics as an initial step in the drug development process.
tial step in the drug development process.  +
Has title ''Drosophila'' as a model to study therapeutic approaches for mitochondrial diseases.  +
MiP area Instruments;methods  + , mtDNA;mt-genetics  + , mt-Medicine  + , Pharmacology;toxicology  +
Stress Mitochondrial disease  +
Was submitted in year 2015  +
Was submitted to event MiP2015 +
Was written by Foriel S + , Smeitink JAM + , Willems PH + , Schenck A + , Beyrath J +
Categories Abstracts
Modification date
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14:17:38, 4 September 2015  +
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